VPS33B mutation with ichthyosis, cholestasis, and renal dysfunction but without arthrogryposis: Incomplete ARC syndrome phenotype
pmid: 16492441
VPS33B mutation with ichthyosis, cholestasis, and renal dysfunction but without arthrogryposis: Incomplete ARC syndrome phenotype
Arthrogryposis-renal dysfunction-cholestasis (ARC) syndrome is a rare multisystem disorder first described in 1979 and recently ascribed to mutation in VPS33B, whose product acts in intracellular trafficking. Arthrogryposis, spillage of various substances in the urine, and conjugated hyperbilirubinemia define an ARC core phenotype, in some patients associated with ichthyosis, central nervous system malformation, deafness, and platelet abnormalities. We describe a patient with cholestasis, aminoaciduria, ichthyosis, partial callosal agenesis, and sensorineural deafness who, although homozygous for the novel VPS33B mutation 971delA/K324fs, predicted to abolish VPS33B function, did not exhibit arthrogryposis. The phenotypes associated with VPS33B mutation may include incomplete ARC.
- King's College London United Kingdom
- San Francisco General Hospital United States
- King's College Hospital United Kingdom
- Kings College London, University of London United Kingdom
- King's College Hospital NHS Foundation Trust United Kingdom
Arthrogryposis, Cholestasis, Hearing Loss, Sensorineural, Vesicular Transport Proteins, 610, Ichthyosis, Infant, Membrane Proteins, Syndrome, Fatal Outcome, Phenotype, 616, Mutation, Humans, Female, Kidney Diseases, Agenesis of Corpus Callosum, Renal Aminoacidurias, Hyperbilirubinemia
Arthrogryposis, Cholestasis, Hearing Loss, Sensorineural, Vesicular Transport Proteins, 610, Ichthyosis, Infant, Membrane Proteins, Syndrome, Fatal Outcome, Phenotype, 616, Mutation, Humans, Female, Kidney Diseases, Agenesis of Corpus Callosum, Renal Aminoacidurias, Hyperbilirubinemia
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